The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility in the pilot phase of performing real-time drug screening on tissue taken during surgery in patients with relapsed medulloblastoma or ependymoma and of having a specialized tumor board assign a treatment plan based on the results of this screening and genomic sequencing. The aim of this trial is to allow every child and young adult with relapsed medulloblastoma and ependymoma to receive the most effective and least toxic therapies currently available and will pave the way for improved understanding and treatment of these tumors in the future. Moreover, if successful, it could serve as a paradigm for personalized medicine programs for other types of cancer.
Participants must have recurrent medulloblastoma or recurrent ependymoma previously histologically confirmed. Participants must be experiencing their first or second relapse to be eligible.
Participants must have surgically accessible disease.
The participant must have received at least one prior therapy at the time of initial diagnosis.
Relapsed medulloblastoma or relapsed ependymoma are eligible.
Participants who have had chemotherapy or radiotherapy within 3 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study or those who have not recovered from adverse events due to agents administered more than 4 weeks earlier.
Participants who are receiving any other investigational agents.
Participants must be at least 7 days since the completion of therapy with a biologic or small molecule agent. For any agent with known adverse events that can occur beyond 7 days after administration, the period prior to enrollment must be beyond the time during which adverse events are known to occur. Such participants should also be discussed with study chairs.
Participants who are currently taking any anti-cancer direct therapy. Steroids are not considered anti-cancer therapy.
Sequential
Specialized Tumor Board Treatment Plan
Other interventionSpecialized Treatment Plan of up to four FDA approved drugs based on participant's screening results will be assigned by PNOC specialized tumor board
Combinations
Other interventionSpecialized Treatment Plan of up to four FDA approved drugs based on participant's screening results will be assigned by PNOC specialized tumor board
Number of participants for whom treatment recommendations are fully completed within 21 business days of tissue collection based on drug screening (Pilot Phase)
Time to tissue collection will be used to determine the feasibility of using the results of real-time in vitro drug screening, WES and RNAseq of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent medulloblastoma. Participants are expected to receive treatment plan within 21 business days
Percentage of participants with treatment recommendations within 21 business days (Pilot Phase)
Time to tissue collection will be used to determine the feasibility of using the results of real-time in vitro drug screening, WES and RNAseq of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent medulloblastoma. Participants are expected to receive treatment plan within 21 business days
Number of participants without adequate tissue
The number of consented participants who do not have adequate tissue collection will be used to determine the feasibility.
Median Time from tissue collection to issued treatment plan from the specialized tumor board (Pilot Phase)
Time to tissue collection will be used to determine the feasibility of using the results of real-time in vitro drug screening, WES and RNAseq of participant-derived specimens to guide treatment recommendations by a specialized tumor board, in a clinically-actionable timeframe, for children and young adults with recurrent medulloblastoma. Participants are expected to receive treatment plan within 21 business days
Proportion of participants with Adverse Events
Proportion of participants with grade 3 or higher toxicities classified according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Adverse events and clinically significant laboratory abnormalities (meeting Grade 3 or higher criteria will be summarized by maximum grade and relationship to study drug(s).
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University of California, San Francisco
Lead sponsor
Washington University School of Medicine
Collaborator
St. Baldrick's Foundation
Collaborator
Ashion Analytics
Collaborator
University of Washington
Collaborator
Pediatric Neuro-Oncology Consortium
Collaborator
